Friday, 6 March 2015

Working with Pharma. Ed Owen's US blog part 2

As well as meeting the
Cystic Fibrosis Foundation in the US last week, the Trust’s Chief Executive, Ed
Owen, and Director of Research & Care, Dr Janet Allen, also dropped in on
two pharmaceutical companies of importance to cystic fibrosis, Novartis and
Vertex. Here is Ed’s second blog explaining what they find out:

Much
is said about the pharmaceutical industry. Some of it is fair, some less so.
But the fact remains that only pharma companies have the size, expertise and
capability to develop and produce the drugs that can make a difference to
people with cystic fibrosis. So it is vital that we have strong relationships
with key companies, like Vertex and Novartis, and work with them for the
benefit of our community.

The
Vertex story is an extraordinary one, and one in which our sister charity, the
Cystic Fibrosis Foundation (CFF), has played a central role. It also tells us
much about the industry, its weaknesses and strengths, limitations and
potential.

Long
frustrated by the lack of industry interest in cystic fibrosis in the late
1990s and early 2000s, the CFF began funding a number of commercial programmes,
including one led by a biotech company called Aurora Sciences, later bought by
Vertex, to find compounds to develop drugs to tackle the genetic causes of
cystic fibrosis.

A
decade and a half on, and Vertex is a pharmaceutical trailblazer. Ivacaftor
(Kalydeco), is transforming the lives
of those with the G551D mutation and a combination therapy of ivacaftor and
lumacaftor has been submitted for approval for use in people with two copies of
the F508del mutation.

Being
a trailblazer however comes at a price, and the high price of ivacaftor has
been controversial – with many questions about the likely price of the
combination therapy if it is granted approval from the regulators.

With
a cash-strapped NHS, and a system of drug appraisal that in England, at least,
is ill-suited to assess particular issues relevant to conditions like cystic
fibrosis, there is a real risk that this combination therapy is given
regulatory approval in Europe later this year for use in four out of 10 people
with cystic fibrosis but will not reach those that need it here in the UK for a
considerable amount of time, if at all.

Our
focus must be to ensure that approved therapies get to those who need them as
quickly as possible - and we are discussing these issues now with all key
players.

But,
when we met them last week, Vertex continued to be tight-lipped about future
pricing in advance of regulatory approval. Along with other pharmaceutical
companies, the company argues that the price of drugs reflects both the high
cost of investment that has been made over many years to develop and trial
therapies (estimates vary, but the average cost of producing a new drug is
usually well above £1bn) and the relatively small numbers of patients with ‘rare
diseases’ like cystic fibrosis.

Like
every other pharmaceutical company, Vertex has shareholders who demand an
appropriate financial return on their investment. The long-term prospects of
the cystic fibrosis ‘market’ means that Vertex is continuing to invest in both
their immediate pipeline of potential therapies and in longer-term research to
develop what they describe as ‘second generation’ drugs that they believe could
be even more effective in future years.

We
are delighted they are doing so, and are keen to see other drugs companies
invest in cystic fibrosis research too. One such company is Novartis. It
already produces cystic fibrosis drugs, most commonly Tobramycin. But we have
had concerns about its future commitment following its decision to move its wider
respiratory research base from Sussex in the UK to Boston in the US in late
2013.

We
were therefore pleased to meet the Novartis team in Boston last week and to
receive their reassurance that their cystic fibrosis-related drug programmes
were alive and well.

For
the first time in decades, the pharmaceutical industry is investing heavily in
cystic fibrosis. As President Obama said in
January,
the condition is at the cutting edge of new advances in ‘precision medicine’
with new therapies being developed focused on defined patient groups with
particular genetic mutations.

That
investment will only continue for as long as companies believe that health
systems will pay for the drugs that follow. Therein lies the challenge for the
NHS, ourselves and all those wishing to see innovative treatments that
transform the lives of people with cystic fibrosis accelerated as quickly as
possible.

High-cost
drugs are the future. How they are paid for is the key question that we all
need to address.